A study to evaluate the pharmacokinetics of Afamelanotide in patients with erythropoietic protoporphyria (EPP).
Summary description of the study
The aim of the CUV052 study is to determine the pharmacokinetics of Afamelanotide in patients with EPP (adolescent and adult patients) and to test the safety and tolerability of Afamelanotide in both adult and adolescent EPP patients. Afamelanotide is a synthetically produced chemical that is very similar to the natural human hormone Alpha-Melanocyte-Stimulating Hormone (α-MSH). Afamelanotide acts similarly to the natural hormone that increases skin melanization. In EPP patients, Afamelanotide activates the production of eumelanin in the melanocytes without prior cell damage in response to UV radiation. This increased production of eumelanin provides EPP patients with light protection, aiming to reduce the number and severity of phototoxic reactions they experience due to this disease while improving their quality of life. SCENESSE® (Afamelanotide 16mg implant) is approved for the treatment of EPP patients aged 18 years and older in the EU, Australia, and the USA. Each patient receives an Afamelanotide implant and is monitored over a period of 90 days. Throughout the study, patients will also undergo safety assessments, including blood and urine analyses, vital signs, and checks of concomitant medication and adverse events.
(BASEC)
Intervention under investigation
SCENESSE® (Afamelanotide 16 mg implant)
(BASEC)
Disease under investigation
Erythropoietic Protoporphyria (EPP)
(BASEC)
• Fourteen adult EPP patients aged between 18 and 70 years (inclusive) and fourteen EPP adolescents aged between 12 and 17 years (inclusive). • >50 kg • Participants are able and willing to comply with the protocol requirements, including refraining from the use of tanning products and excessive exposure to ultraviolet (UV) light from the start of the study until day 90. (BASEC)
Exclusion criteria
• Significant history of allergies and/or sensitivity to any of the components of the investigational medicinal product. • Administration of Afamelanotide in the last 60 days • Donation of 400 ml or more of blood or significant blood loss in the eight weeks prior to screening. (BASEC)
Trial sites
Zurich
(BASEC)
Sponsor
CLINUVEL AG
(BASEC)
Contact
Contact Person Switzerland
Head of Clinical Operations
+44 1372 860 765
mail@clutterclinuvel.comCLINUVEL UK
(BASEC)
Scientific Information
not available
Name of the authorising ethics committee (for multicentre studies, only the lead committee)
Ethics Committee Zurich
(BASEC)
Date of authorisation
12.03.2024
(BASEC)
ICTRP Trial ID
not available
Official title (approved by ethics committee)
A Study to Evaluate the Pharmacokinetics of Afamelanotide in Patients with Erythropoietic Protoporphyria (EPP) (BASEC)
Academic title
not available
Public title
not available
Disease under investigation
not available
Intervention under investigation
not available
Type of trial
not available
Trial design
not available
Inclusion/Exclusion criteria
not available
not available
Primary and secondary end points
not available
not available
Registration date
not available
Incorporation of the first participant
not available
Secondary sponsors
not available
Additional contacts
not available
Secondary trial IDs
not available
Results-Individual Participant Data (IPD)
not available
Further information on the trial
not available
Results of the trial
Results summary
cuv052-laypersons-summary-of-results-de.pdfLink to the results in the primary register
not available